Latest Inborn errors of metabolism Stories
Cells, like ordinary households, produce "garbage" â€“ debris and dysfunctional elements â€“ that need disposal.
Cells, like ordinary households, produce "garbage" â€“ debris and dysfunctional elements - that need disposal.
WEINHEIM, Germany, May 17, 2011 /PRNewswire/ -- International biotechnology firm Cytonet, which is investigating a liver cell infusion of donated human liver cells for the treatment of urea cycle disorders (UCD) in young children, announced it has been granted orphan drug designation by the U.S.
CRANBURY, N.J., May 4, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD), a biopharmaceutical company at the forefront of developing therapies for rare diseases, today announced financial results for the first quarter ended March 31, 2011.
Childrenâ€™s Memorial Hospital in Chicago is one of only 12 sites across the United States enrolling patients in a national clinical research trial which, for the first time is the United States, is using an investigational liver cell infusion to treat urea cycle disorders (UCDs) in children. UCDs are congenital and often life-threatening disorders of ammonia metabolism in the liver.
WEINHEIM, Germany, April 14, 2011 /PRNewswire/ -- International biotechnology firm Cytonet recently launched the first ever clinical trial in the United States using an investigational liver cell infusion to treat urea cycle disorders (UCD) in children.
Children with inborn errors of metabolism received vaccines on the same immunization schedule as did healthy infants.
SAN DIEGO, April 7, 2011 /PRNewswire/ -- Zacharon Pharmaceuticals, Inc. today announced that the company has entered into a strategic research collaboration with Pfizer Inc. to develop drugs for orphan diseases, including lysosomal storage disorders.
CRANBURY, N.J., March 8, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND).
CRANBURY, N.J., Feb.